On October 29, 2020 Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported financial results and provided a business update for the third quarter ended September 30, 2020 (Press release, Blueprint Medicines, OCT 29, 2020, View Source [SID1234569377]).

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"Following our tremendous progress in the third quarter, Blueprint Medicines is in a very strong position, with two precision therapies now approved and significant near-term growth opportunities expected across our portfolio," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In recent months, we bolstered our commercial foundation with the U.S. approval and launch of GAVRETO, and we are now preparing to file our supplemental NDA for AYVAKIT in advanced systemic mastocytosis, marking an important step toward delivering a much-needed new therapy to patients across the systemic mastocytosis disease spectrum. In parallel, we continued to strengthen our pipeline with the presentation of compelling preclinical proof-of-concept data for BLU-945, a potentially best-in-class therapy for treatment-resistant EGFR-driven lung cancer, that support our plan to initiate clinical development of BLU-945 in the first half of next year."

Third Quarter 2020 Highlights and Recent Updates

AYVAKIT (avapritinib): systemic mastocytosis (SM)

●Announced positive top-line results from the Phase 1 EXPLORER and Phase 2 PATHFINDER clinical trials of AYVAKIT in patients with advanced SM. Consistent with previously reported EXPLORER trial results, the data showed profound reductions in mast cell burden, high overall response and complete remission rates, and durable clinical benefit, including prolonged median overall survival. AYVAKIT was generally well-tolerated, with an improved safety profile at the 200 mg once daily dose. Read the press release here.

AYVAKIT/AYVAKYT (avapritinib): gastrointestinal stromal tumor (GIST)

●Recorded $6.1 million in net product revenue during the third quarter of 2020 for AYVAKIT, which was approved by the U.S. Food and Drug Administration (FDA) in January 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.
●Received European Commission (EC) conditional marketing authorization for avapritinib under the brand name AYVAKYT as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST harboring the PDGFRA D842V mutation and initiated the first commercial launch following EC approval in Germany. Read the press release here.

GAVRETO (pralsetinib): RET-altered cancers

●Received FDA approval of GAVRETO for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test and launched GAVRETO together with Genentech, a member of the Roche Group, in the U.S. in September. Read the press release here. Please click here to see the full Prescribing Information for GAVRETO and visit www.GAVRETO.com for more information on GAVRETO.
●Reported updated data from the ongoing ARROW clinical trial of GAVRETO in advanced RET-mutant medullary thyroid cancer (MTC) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress

2020. The data showed durable responses and a well-tolerated safety profile for GAVRETO in patients with advanced RET-mutant MTC, with consistent clinical activity in patients across lines of therapy and regardless of RET mutation genotypes. Read the press release here.
●Announced acceptance of U.S. marketing application for GAVRETO for the treatment of patients with advanced or metastatic RET-mutant MTC and RET fusion-positive thyroid cancer. The FDA accepted the new drug application (NDA) for priority review under its Real-Time Oncology Review (RTOR) pilot program, and set an action date of February 28, 2021 under the Prescription Drug User Fee Act.

BLU-945: EGFR-mutated NSCLC

●Reported preclinical proof-of-concept data for BLU-945 at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020. The data showed BLU-945 potently and selectively inhibited triple-mutant epidermal growth factor receptor (EGFR) harboring the most common on-target resistance mutations to standard treatments for EGFR-mutated NSCLC, resulting in robust anti-tumor activity in multiple lung cancer models. Read the press release here.
●Announced plans to develop BLU-945 as a monotherapy and in combination with other agents for the treatment of patients with treatment-resistant EGFR NSCLC.

Corporate:

●Strengthened the company’s leadership team with the appointment of Fouad Namouni, M.D., as President, Research & Development. In this new role, Dr. Namouni leads a joint research and development organization, overseeing all phases of product development from discovery through global registration under a unified portfolio vision.

Key Upcoming Milestones

The company expects to achieve the following near-term milestones:

●Submit a supplemental new drug application to the FDA for avapritinib for the treatment of patients with advanced SM in the fourth quarter of 2020.
●Obtain U.S. approval of GAVRETO for RET-altered thyroid cancers in the first quarter of 2021.
●Initiate a global Phase 1 dose-escalation trial for BLU-945 in EGFR-mutated NSCLC in the first half of 2021.

Third Quarter 2020 Financial Results

●Revenues: Revenues were $745.1 million for the third quarter of 2020, including $6.1 million of net product revenues from sales of AYVAKIT, $0.2 million of net product revenues from sales of GAVRETO and $738.8 million in collaboration revenues under the collaboration agreements with Roche and CStone. Blueprint Medicines recorded $9.1 million in collaboration revenues for the third quarter of 2019.
●Cost of Sales: Cost of sales was $0.1 million for the third quarter of 2020. Blueprint Medicines did not incur cost of sales in the third quarter of 2019, as no product sales were generated during that period.
●R&D Expenses: Research and development expenses were $74.2 million for the third quarter of 2020, as compared to $81.5 million for the third quarter of 2019. This decrease was primarily due to reimbursement from the global development cost sharing arrangement under the collaboration agreement with Roche for pralsetinib. Research and development expenses included $8.6 million in stock-based compensation expenses for the third quarter of 2020.
●SG&A Expenses: Selling, general and administrative expenses were $37.4 million for the third quarter of 2020, as compared to $25.6 million for the third quarter of 2019. This increase was primarily due to an increase in costs and personnel expenses associated with building Blueprint Medicines’ commercial infrastructure for commercialization of AYKAKIT and GAVRETO, partially offset by reimbursement for Roche’s share of the loss generated from the commercialization of GAVRETO in the U.S. under the collaboration for pralsetinib. Selling, general and administrative expenses included $11.0 million in stock-based compensation expenses for the third quarter of 2020.

●Net Income (Loss): Net income was $634.0 million for the third quarter of 2020, or a diluted net income per share of $11.16, as compared to a net loss of $94.3 million for the third quarter of 2019, or a diluted net loss per share of $1.93.
●Cash Position: As of September 30, 2020, cash, cash equivalents and investments were $1,355.9 million, as compared to $548.0 million as of December 31, 2019. This increase was primarily related to upfront payments of $775.0 million received in the third quarter of 2020 under Blueprint Medicines’ collaboration with Roche and $308.4 million in net proceeds received from Blueprint Medicines’ January 2020 follow-on underwritten public offering, partially offset by cash used in operating activities.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss third quarter 2020 financial results and recent business activities. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international), and referring to conference ID 2995408. A webcast of the call will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conference

Blueprint Medicines will participate in the Jefferies Virtual London Healthcare Conference on November 17, 2020 at 2:55 p.m. ET. A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.